A phase 3 trial highlights betibeglogene autotemcel as a potentially curative gene therapy for severe transfusion-dependent β ...

While gene therapy has emerged as a promising alternative for treating beta-thalassemia, hematopoietic stem cell ...

Aruvant is developing ARU-1801, a one-time, potentially curative gene therapy for sickle cell disease and β-thalassemia. In an ongoing clinical phase 1/2 study, ARU-1801, administered with only ...

Earlier this year, the US Food and Drug Administration (FDA) approved the first potentially curative gene therapy to treat beta-thalassemia. While multiple companies have been working to bring ...

who is now able to hear thanks to gene therapy ... approval of a CRISPR therapy designed for patients suffering from sickle cell anemia and the blood disorder beta thalassemia.

In a world first, medical regulators in the UK have approved a gene therapy that aims to cure two blood disorders. The treatment for sickle cell disease and beta thalassemia is the first to be ...

More information: Maxwell Elliott Chappell et al, Use of HSC Targeted LNP to Generate a Mouse Model of Lethal α-Thalassemia and Treatment via Lentiviral Gene Therapy, Blood Journal (2024).